The Efficacy of Pharmacological Interventions in Pulmonary Arterial Hypertension (WHO Group 1): A Systematic Review of Randomized Controlled Trials
Abstract
Introduction: Pulmonary arterial hypertension (PAH), WHO Group 1, is a progressive vasculopathy leading to right heart failure and premature death. Pharmacological therapies targeting the endothelin, nitric oxide, and prostacyclin pathways have improved outcomes, but the optimal treatment strategy remains a subject of ongoing investigation. This systematic review synthesizes evidence from randomized controlled trials (RCTs) to evaluate the efficacy of these interventions, with a focus on the comparative benefits of monotherapy versus combination therapy.
Methods: A systematic search of MEDLINE, Embase, the Cochrane Central Register of Controlled Trials, and ClinicalTrials.gov was conducted for RCTs published from January 1990 to the present. Studies enrolling adult patients with WHO Group 1 PAH and evaluating approved pharmacological agents against placebo or another active therapy were included. Data on study design, patient characteristics, and a minimum of 15 predefined outcomes—including functional, hemodynamic, biomarker, and clinical event endpoints—were extracted. Methodological quality was assessed using the Cochrane Risk of Bias 2 (RoB 2) tool. A narrative synthesis was performed.
Results: Seventeen pivotal RCTs were included in the final analysis. Monotherapy with endothelin receptor antagonists (ERAs), nitric oxide pathway modulators (phosphodiesterase-5 inhibitors and soluble guanylate cyclase stimulators), and prostacyclin pathway agents demonstrated significant…
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